Trial of Cyclosporine in the Acute Phase of Leber Hereditary Optic Neuropathy

University Hospital, Angers12 enrolled

Overview

The Leber Hereditary Optic Neuropathy is a genetic disorder caused by maternal transmission of mitochondrial DesoxiroboNucleid Acid mutations. It is manifested by a rapidly progressive blindness, profound, due to atrophic optic nerve. The visual loss is primarily unilateral bilateralisation taking place in the vast majority of cases in weeks or months. The neuro-cardio-protective properties of cyclosporine (and its analogs specifically targeting the anti-apoptotic mechanisms) are particularly promising. The investigators hypothesis is that cyclosporine may limit apoptosis during the acute phase of the disease process and would limit the loss of visual acuity and improve the visual prognosis of these patients.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Leber Hereditary Optic Neuropathy

Interventions

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * patient with the mutation confirmed by molecular analysis * patient with a recent loss of monocular vision (≤ 6 months) * voluntarily Patient Consent Exclusion Criteria: * patient who have not given their written and informed consent signed * against indication of cyclosporine * no drug compliance to previous inclusion * no national health insurance affiliation * pregnant women or lactating * women who could become pregnant during the study period and with no contraception * private patients of their liberty by judicial or administrative decision, or patients under supervision

Trial of Cyclosporine in the Acute Phase of Leber Hereditary Optic Neuropathy

Overview

Conditions

Interventions

Eligibility

Locations (1)

Outcomes

Primary Outcomes

Central Contacts